If you or your child are participating in an early phase of a clinical trial, you likely will not be allowed to continue the treatment being tested. Researchers will need to analyze the study results to ensure that the treatment is safe, effective, and better than current treatments. It would not make sense to continue the potential treatment until this process can be completed.
Open-Label Use
Some clinical trials do have what is called an open-label extension study, which means the doctors and participants know what treatment is being provided. This study typically occurs after a Phase 3 clinical trial of a new treatment. Participants are invited to enroll, so that additional safety information can be gathered about long-term use while the U.S. Food and Drug Administration (FDA) reviews the treatment for approval.
Expanded Access
The study sponsor can provide expanded access (sometimes called compassionate use) to a new treatment to patients with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial. FDA regulations allow manufacturers to provide the drug as long as it does not pose unreasonable risks to the patients, and the patients do not have any other options.
Approved Drug Being Tested for CF
Some cystic fibrosis clinical trials test drugs that the FDA already has approved for another use. If the FDA has already approved the drug, then your CF doctor can prescribe it to you after your participation in the trial has ended.
How the Pipeline Fits In
The Cystic Fibrosis Foundation maintains a robust pipeline of therapies that target CF from every angle. The progress of these therapies can be tracked by using our interactive Drug Development Pipeline tool.
The pipeline shows each major category of therapies that the Foundation has helped either financially or through clinical trials within the Therapeutics Development Network. Although the pipeline is not a comprehensive list of CF therapies in clinical trials, it provides a snapshot of the therapeutic areas that the Foundation is pursuing.